Participation of Somatic Stem Cells, Labeled by a Unique …

A monoclonal antibody (A3) was generated by using rat malignant fibrous histiocytoma (MFH) cells as the antigen. Generally, MFH is considered to be a sarcoma derived from undifferentiated mesenchymal cells. Molecular biological analyses using the lysate of rat MFH cells revealed that A3 is a conformation specific antibody recognizing both N-glycan and peptide. A3-labeled cells in bone marrow were regarded as somatic stem cells, because the cells partly coexpressed CD90 and CD105 (both immature mesenchymal markers). In the hair follicle cycle, particularly the anagen, the immature epithelial cells (suprabasal cells) near the bulge and some immature mesenchymal cells in the disassembling dermal papilla and regenerating connective tissue sheath/hair papilla reacted to A3. In the cutaneous wound-healing process, A3-labeled epithelial cells participated in re-epithelialization in the wound bed, and apparently, the labeled cells were derived from the hair bulge; in addition, A3-labeled immature mesenchymal cells in the connective tissue sheath of hair follicles at the wound edge showed the expansion of the A3 immunolabeling. A3-labeled immature epithelial and mesenchymal cells contributed to morphogenesis in the hair cycle and tissue repair after a cutaneous wound. A3 could become a unique antibody to identify somatic stem cells capable of differentiating both epithelial and mesenchymal cells in rat tissues.

Keywords: N-glycan; antibody; cutaneous wound healing; hair follicle cycle; somatic stem cells.

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Participation of Somatic Stem Cells, Labeled by a Unique ...

COVID-19 Impact on Global Cell Therapy Industry 2020: Market Trends, Size, Share, Applications, SWOT Analysis by Top Key Players and Forecast Report…

TheGlobal Cell Therapy Marketwas estimated to be valued at USD XX million in 2018 and is projected to reach USD XX million by 2026, at a CAGR of XX% during 2019 to 2026. Growing aging patient population, the rise in cell therapy transplantations globally, and rising disease awareness drive the growth of the market.

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Cell therapyinvolves the administration of somatic cell preparations for the treatment of diseases or traumatic damages. The objective of this study is to provide long term treatment through a single injection of therapeutic cells.

However, stringent regulatory policies may restrain growth of the market in the forecast period.

The global Cell Therapy Market is primarily segmented based on different type, technique, cell source, technology, end users and region.

On the basis of type, the market is split into:* Allogenic Therapies* Autologous Therapies

On the basis of technique, the market is split into:* Stem Cell Therapy* Cell Vaccine* Adoptive Cell Transfer (ACT)* Fibroblast Cell Therapy* Chondrocyte Cell Therapy* Other Technique

On the basis of cell source, the market is split into:* Bone Marrow* Adipose Tissue* Umbilical Cord Blood-Derived Cells

On the basis of technology, the market is split into:* Viral Vector Technology* Cell Immortalization Technology* Genome Editing Technology

On the basis of end user, the market is split into:* Hospitals & Clinics* Regenerative Medicine Centers* Other End Users

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Moreover, the market is classified based on regions and countries as follows:* North America- U.S., Canada* Europe- U.K., France, Germany, Italy and Rest of Europe* Asia-Pacific- China, Japan, India and Rest of Asia Pacific* South America- Brazil, Mexico and Rest of South America* Middle East & Africa- South Africa, Saudi Arabia and Rest of Middle East & Africa

Key Market Players: The key players profiled in the market include:* Kolon TissueGene, Inc.* JCR Pharmaceuticals Co. Ltd.* Osiris Therapeutics, Inc.* Stemedica Cell Technologies, Inc.* Fibrocell Science, Inc.* Vericel Corporation* Pharmicell Co., Ltd* Anterogen.CO.,LTD.* Baxter Healthcare Corporation.* Arteriocyte Medical Systems

These enterprises are focusing on growth strategies, such as new product launches, expansions, acquisitions, and agreements & partnerships to expand their operations across the globe.

Key Benefits of the Report:* Global, regional, country, type, technique, cell source, technology, end users market size and their forecast from 2018-2026* Identification and detailed analysis on key market dynamics, such as, drivers, restraints, opportunities, and challenges influencing growth of the market* Detailed analysis on industry outlook with market specific PESTLE, and supply chain to better understand the market and build expansion strategies* Identification of key market players and comprehensively analyze their market share and core competencies, detailed financial positions, key product, and unique selling points* Analysis on key players strategic initiatives and competitive developments, such as joint ventures, mergers, and new product launches in the market* Expert interviews and their insights on market shift, current and future outlook, and factors impacting vendors short term and long term strategies* Detailed insights on emerging regions, type, technique, cell source, technology and end users with qualitative and quantitative information and facts

Target Audience:* Cell Therapy Manufactures* Traders, Importers, and Exporters* Raw Material Suppliers and Distributors* Research and Consulting Firms* Government and Research Organizations* Associations and Industry Bodies

Research Methodology:The market is derived through extensive use of secondary, primary, in-house research follows by expert validation and third party perspective, such as, analyst reports of investment banks. The secondary research is the primary base of our study wherein we conducted extensive data mining, referring to verified data products, such as, white papers, government and regulatory published articles, technical journals, trade magazines, and paid data products.

For forecasting, regional demand & supply factors, recent investments, market dynamics including technical growth scenario, consumer behavior, and end use trends and dynamics, and production capacity were taken into consideration. Different weightages have been assigned to these parameters and quantified their market impacts using the weighted average analysis to derive the market growth rate.

The market estimates and forecasts have been verified through exhaustive primary research with the Key Industry Participants (KIPs), which typically include:* Manufacturers* Suppliers* Distributors* Government Body & Associations* Research Institutes

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Table of Content1. Introduction2. Research Methodology3. Executive Summary4. Global Cell Therapy Market Overview4.1. Market Segmentation & Scope4.2. Market Trends4.2.1. Drivers4.2.2. Restraints4.2.3. Opportunities4.2.4. Supply Chain Analysis4.3. Global Cell Therapy Market Porters Five Forces Analysis4.4. Global Cell Therapy Market PESTEL Analysis5. Global Cell Therapy Market, by Type5.1. Global Cell Therapy Market, Size and Forecast, 2015-20265.2. Global Cell Therapy Market, by Allogenic Therapies, 2015-20265.2.1. Key driving factors, trends and opportunities5.2.2. Market size and forecast, 2015-20265.3. Global Cell Therapy Market, by Autologous Therapies, 2015-20265.3.1. Key driving factors, trends and opportunities5.3.2. Market size and forecast, 2015-20266. Global Cell Therapy Market, by Technique6.1. Global Cell Therapy Market, Size and Forecast, 2015-20266.2. Global Cell Therapy Market, by Stem Cell Therapy, 2015-20266.2.1. Key driving factors, trends and opportunities6.2.2. Market size and forecast, 2015-20266.3. Global Cell Therapy Market, by Cell Vaccine, 2015-20266.3.1. Key driving factors, trends and opportunities6.3.2. Market size and forecast, 2015-2026

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FDA Approves Elixirgen Therapeutics IND Application for Therapy for Telomere Biology Disorders with Bone Marrow Failure – Herald-Mail Media

BALTIMORE, June 4, 2020 /PRNewswire/ -- Elixirgen Therapeutics, Inc., a Baltimore-based biotechnology company focused on the discovery, development, and commercialization of therapies for genetic diseases and vaccines, received confirmation from the U.S. Food and Drug Administration (FDA) that its Investigational New Drug (IND) application for its lead candidate, EXG34217, was approved on May 23, 2020. EXG34217 is an autologous cell therapy for telomere biology disorders with bone marrow failure.

The FDA's approval allows Elixirgen Therapeutics to proceed with its planned Phase I/II, open label, single center clinical trial to assess the safety and tolerability of EXG34217 at Cincinnati Children's Hospital Medical Center (ClinicalTrials.gov Identifier: NCT04211714). This program's treatment paradigm uses Elixirgen Therapeutics' proprietary ZSCAN4 technology to extend the telomeres of the hematopoietic stem cells of the patients.

About Elixirgen Therapeutics, Inc.Elixirgen Therapeutics, Inc. is a Baltimore-based biotechnology company co-founded by Akihiro Ko and Minoru Ko, MD, PhD, which is focused on curing humanity's ailments through innovations in stem cell biology.The company's experienced team of researchers has a wide variety of specialties, enabling it to use both basic and translational research approaches to developing therapies for genetic diseases and vaccines. For more information visit https://ElixirgenTherapeutics.com

Forward-Looking StatementsThis press release may contain "forward-looking" statements, including statements regarding the effectiveness of EXG34217 to treat telomere biology disorders with bone marrow failure and statements relating to the planned clinical trials of EXG34217. Actual results may differ materially from those set forth in this press release due to the risks and uncertainties inherent in drug research and development. In light of these and other uncertainties, the forward-looking statements included in this press release should not be regarded as a representation by Elixirgen Therapeutics that its plans and objectives regarding EXG34217 will be achieved. Any forward-looking statements in this press release speak only as of the date of this press release, and Elixirgen Therapeutics undertakes no obligation to update or revise the statementsin the future, even if new information becomes available.

Contact:Media RelationsElixirgen Therapeutics, Inc.(443) 869-5420Media@ElixirgenTherapeutics.com

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FDA Approves Elixirgen Therapeutics IND Application for Therapy for Telomere Biology Disorders with Bone Marrow Failure - Herald-Mail Media

Global and United States Adipose Derived Stem Cell Therapy Market 2020 (Coronavirus (COVID-19) Business Impact and Analysis) | Research Industry – SG…

The report study researched by Research Industry US gives comprehensive knowledge and valuable insights about the Global and United States Adipose Derived Stem Cell Therapy Market. Also, the study attempts to deliver significant and detailed insights into the current market prospect and emerging growth scenarios. The report on the Global and United States Adipose Derived Stem Cell Therapy Market also emphasizes on market players as well as the new entrants in the market landscape.

Global and United States Adipose Derived Stem Cell Therapy Market is estimated to reach $XX billion in 2019 with a CAGR of XX% from 2019 to 2025.

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The Global and United States Adipose Derived Stem Cell Therapy Market report is a precise and deep-dive study on the current state that aims at the major drivers, market strategies, and imposing growth of the key players. Worldwide Adipose Derived Stem Cell Therapy Market Industry also offers a granular study of the dynamics, segmentation, revenue, share forecasts, and allows you to make superior business decisions. The report serves imperative statistics on the market stature of the prominent manufacturers and is an important source of guidance and advice for companies and individuals involved in the Adipose Derived Stem Cell Therapy Market industry.

Impact of Coronavirus (COVID-19) on Global and United States Adipose Derived Stem Cell Therapy Market.

Coronavirus (COVID-19) is spreading across the world with a serious impact on the economy and the global market. The report considers and accounts for the impact of COVID-19 on Global and United States Adipose Derived Stem Cell Therapy Market across all the segments, regions, countries, and key players. North America and Europe are worst-hit countries by Coronavirus which are key players in the global economy. The report provides a detailed analysis of the impact on the market, growth strategies, supply china disruption, consumption pattern of the Global and United States Adipose Derived Stem Cell Therapy Market.

The report provides market size with 2019 as the base year in consideration and a yearly forecast until 2026 in terms of Revenue (USD Million). The estimates for all segments including type and application have been provided on a regional basis for the forecast period mentioned above. We have implemented a mix of top-down and bottom-up approaches for market sizing, analyzing the key regional markets, dynamics, and trends for various applications.

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The Global and United States Adipose Derived Stem Cell Therapy Market has been estimated by integrating the regional markets.

By Type:

Cells, Allogeneic Stem Cells, etc.

By Application:

Application, Research Application, etc.

Competitive Landscape:

Key players profile in the report include

Market segment by players, this report coversAlloCureMesoblastCelllerisAntriaIntrexonCelgene CorporationTissue GenesisCytori TherapeuticsCorestemPluristem TherapeuticsCyagenBioRestorative TherapiesLonzaPluristem TherapeuticsCelltex Therapeutics CorporationiXCells Biotechnologies

The report of Global and United States Adipose Derived Stem Cell Therapy Market studies the key players present in the market. The chapter includes the competitive landscape section which provides the full and in-depth analysis of the current market trends, changing technologies, and developments that will be beneficial for the companies, which are competing in the market. The report offers an overview of revenue, demand, and supply of data, futuristic cost, and growth analysis during the projected year. In addition to a brief overview of the company, analysts shed light on their valuation and evolution. It also discusses the list of important products and the ones in the pipeline. The competitive landscape is analyzed by understanding the approaches of the companies and the initiatives they have taken in recent years to triumph over the intensive competition.

A section of the report has given complete information about regional analysis. It provides a market outlook and positions the forecast within the context of the overall Global and United States Adipose Derived Stem Cell Therapy Market. Research Industry US has segmented the Global and United States Adipose Derived Stem Cell Therapy Market into major geographical regions such as North America, Europe, Asia Pacific, South America, and the Middle East and Africa. Potential new entrants desiring to target only high growth areas are also incorporated in this informative section of the Global and United States Adipose Derived Stem Cell Therapy Market.

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Report Objectives:

Get In Touch!Research IndustryNavale ICON IT Park,Office No. 407, 4th Floor, Mumbai Banglore Highway, Narhe, PuneMaharashtra 411041Phone +1 213-275-4706Emailsales@researchindustry.us

David Barstow is a senior editor at SG Research Sphere covering products, apps, services, and consumer tech issues and trends. He is very active in social media and collects regular information for the company. He brings a great vision to the nexus of content and the social, digital, video and pragmatic network. He has eight years of experience with his expertise in conglomerate industries.

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Global and United States Adipose Derived Stem Cell Therapy Market 2020 (Coronavirus (COVID-19) Business Impact and Analysis) | Research Industry - SG...

MSC Therapy for Acute Respiratory Distress Syndrome; It’s Time to Accelerate Clinical Trials for COVID-19 Patients in Need – P&T Community

WASHINGTON, June 3, 2020 /PRNewswire/ --A new systematic review and meta-analysis of clinical studies using mesenchymal stromal cells (MSCs) led by a team at the Mayo Clinic, and including researchers from Emory, Duke, Case-Western, and the University of Miami, shows a trend toward improved outcomes and reduced mortality for patients with acute respiratory distress syndrome (ARDS), a major complication for patients with COVID-19. This studyand several othersalso have shown that MSCs are safe for patients.

Based on these findings, the authors call for the rapid commencement of large-scale, confirmatory clinical trials to build on the existing evidence base, which shows a trend toward improved pulmonary function and reduced severe lung inflammation for patients with ARDS, paving the way toward another treatment option for seriously ill patients with COVID-19.

To date, nearly two million Americans have tested positive for COVID-19 and more than 100,000 Americans have died. In its most severe form, COVID-19 leads to ARDSa life-threatening lung injury that allows fluid to leak into the lungs and makes it difficult for patients to breathe. More than 40 percent of individuals hospitalized for severe and critical COVID-19 develop ARDS, and 22 percent to 62 percent of those who are diagnosed and become critically ill, die from the disease. There is no effective treatment for ARDS today; MSCs potentially offer a unique therapeutic option to help patients in need.

"The analysis shows a positive trend in outcomes when treating ARDS patients with MSC therapy and represents the potential to save thousands of patients with COVID-19 induced ARDS," said Wenchun Qu, MD, PhD of the Mayo Clinic and first author of the paper. "The potential benefitcombined with the demonstrated safety of these therapiessupports the need for rapid commencement of more clinical trials."

"Acute respiratory distress syndrome is a rapidly progressive disease that can occur in critically ill patients," said Anthony Atala, MD, Editor-in-Chief of STEM CELLS Translational Medicine and director of the Wake Forest Institute for Regenerative Medicine. "Having additional potential therapies, such as MSCs, could be highly beneficial to patients with COVID-19."

To date, the FDA has approved more than a dozen investigational new drug applications for the use of MSCs for COVID-19-related conditions. The National Institutes of Health (NIH) has also supported the use of MSCs and other regenerative cell therapies to help patients with other conditions. The bipartisan 21st Century Cures Act provided $30 million in funding to the NIH over three years for clinical research for such therapies. However, these limited investments expire in fiscal year 2020.

The Alliance for Cell Therapy Now and the Regenerative Medicine Foundation support the recommendation of the authors, who urge funding for larger studies that build on the results to date. Collaboration and funding are also needed to collect and analyze the evidence from multiple ongoing and new studies, to better evaluate outcomes and potential benefits of MSC therapy for COVID-19 patients in need. A portion of the more than $10 billion in funding directed by Congress to the Biomedical Advanced Research and Development Authority (BARDA) and the NIH for COVID-19 should be used to support these goals.

About the Alliance for Cell Therapy Now

Alliance for Cell Therapy Now (ACT Now) is an independent, non-profit organization devoted to advancing the availability of and access to safe and effective cell therapies for patients in need. ACT Now convenes experts and stakeholders to develop and advance sound policies that will improve the development, manufacturing, delivery, and improvement of regenerative cell therapies. See http://allianceforcelltherapynow.org/

About the Regenerative Medicine Foundation

The non-profit Regenerative Medicine Foundation (RMF) fosters strategic collaborations to accelerate the development of regenerative medicine to improve health and deliver cures. RMF pursues its mission by producing its flagship World Stem Cell Summit, honoring leaders through the Stem Cell and Regenerative Medicine Action Awards, and promoting educational initiatives. STEM CELLS Translational Medicine is RMF's official journal partner. See https://www.regmedfoundation.org/

View original content:http://www.prnewswire.com/news-releases/msc-therapy-for-acute-respiratory-distress-syndrome-its-time-to-accelerate-clinical-trials-for-covid-19-patients-in-need-301070329.html

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MSC Therapy for Acute Respiratory Distress Syndrome; It's Time to Accelerate Clinical Trials for COVID-19 Patients in Need - P&T Community

Using big data approaches to develop cell therapies – Drug Target Review

An area where stem cell biology and medicine are combining effectively is the establishment of new cell therapies. However, current therapies are limited to a narrow set of cell types that can be isolated or created and expanded in vitro. Dr Owen Rackham discusses how utilising computational approaches will further enhance applications of stem-cell-derived therapies in the future.

For decades (or perhaps centuries) the approach in cell biology has remained relatively unchanged. We isolate cells and with our confined knowledge of their endogenous conditions, begin to experiment until we can sustain them in vitro. Once established, we can conduct further investigation to assess a cells response to different conditions, changes over time or response to manipulation. This is especially true of stem cell biology, established from tireless efforts to incrementally improve culture conditions or differentiation protocols based on fragmented knowledge of developmental processes. Despite this, the promise of stem-cell therapies is already being realised in the clinic, but the breadth of cell types being used is still relatively narrow. Recent technological advances in the field have been focused on the safe and scalable manufacture of therapies. While these are revolutionary breakthroughs, the applications are largely limited to T cells, haematopoietic- and pluripotent-stem cells (HSCs and PSCs), a small fraction in the grand heterogeneity of cell types. Consequently, the lack of cell source diversity prevents cell therapy from fulfilling its clinical potential, pointing to the need for new means to isolate or generate source cells.

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Using big data approaches to develop cell therapies - Drug Target Review

Miracle cures and fast-track approval threatens medical advances, say scientists – The University of Manchester

Prof. Volker ter Meulen, Co-chair of the EASAC-FEAM Working Group and Past President of EASAC, said: Stem cell and gene-based therapies hold great medical promises. But we are alarmed over a trend to lower requirements of evidence. Also, we see an increasing problem of commercial clinics offering unregulated products and services

The Academies say the idea of regenerative medicine is to tackle diseases which up to now are incurable. According to the report, cosmetic applications, for example, are inappropriate for the time being.

So far, regenerative medicine has proven itself only in few specific clinical indications, for example for skin disorders. Yet, we see an increasing number of unregulated clinics promising a wide range of benefits on the basis of poorly characterised medicinal products with little evidence of effectiveness. They usually advertise their services via the internet with the primary intention of financial profit, explains EASAC Biosciences Programme Director Dr. Robin Fears.

The scientists urge the EU to resist the pressure and put patients first. When countries lower regulatory standards in their eagerness to support national economic interests, it is even more important for the EU as a major global player to defend the principles of international cooperation in health regulation, says Prof. George Griffin, Co-chair of the Working Group and President of FEAM.

We all want cures to be available in the shortest time frame possible. But our analysis and recommendations aim at ensuring that regulatory procedures are robust, transparent and evidence-based, concludes Cossu. Scientific research and proof are more important than ever. The EU and national regulators should be wary of not undermining public trust in science.

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Miracle cures and fast-track approval threatens medical advances, say scientists - The University of Manchester

MSC Therapy for Acute Respiratory Distress Syndrome; It’s Time to Accelerate Clinical Trials for COVID-19 Patients in Need – PRNewswire

WASHINGTON, June 3, 2020 /PRNewswire/ --A new systematic review and meta-analysis of clinical studies using mesenchymal stromal cells (MSCs) led by a team at the Mayo Clinic, and including researchers from Emory, Duke, Case-Western, and the University of Miami, shows a trend toward improved outcomes and reduced mortality for patients with acute respiratory distress syndrome (ARDS), a major complication for patients with COVID-19. This studyand several othersalso have shown that MSCs are safe for patients.

Based on these findings, the authors call for the rapid commencement of large-scale, confirmatory clinical trials to build on the existing evidence base, which shows a trend toward improved pulmonary function and reduced severe lung inflammation for patients with ARDS, paving the way toward another treatment option for seriously ill patients with COVID-19.

To date, nearly two million Americans have tested positive for COVID-19 and more than 100,000 Americans have died. In its most severe form, COVID-19 leads to ARDSa life-threatening lung injury that allows fluid to leak into the lungs and makes it difficult for patients to breathe. More than 40 percent of individuals hospitalized for severe and critical COVID-19 develop ARDS, and 22 percent to 62 percent of those who are diagnosed and become critically ill, die from the disease. There is no effective treatment for ARDS today; MSCs potentially offer a unique therapeutic option to help patients in need.

"The analysis shows a positive trend in outcomes when treating ARDS patients with MSC therapy and represents the potential to save thousands of patients with COVID-19 induced ARDS," said Wenchun Qu, MD, PhD of the Mayo Clinic and first author of the paper. "The potential benefitcombined with the demonstrated safety of these therapiessupports the need for rapid commencement of more clinical trials."

"Acute respiratory distress syndrome is a rapidly progressive disease that can occur in critically ill patients," said Anthony Atala, MD, Editor-in-Chief of STEM CELLS Translational Medicine and director of the Wake Forest Institute for Regenerative Medicine. "Having additional potential therapies, such as MSCs, could be highly beneficial to patients with COVID-19."

To date, the FDA has approved more than a dozen investigational new drug applications for the use of MSCs for COVID-19-related conditions. The National Institutes of Health (NIH) has also supported the use of MSCs and other regenerative cell therapies to help patients with other conditions. The bipartisan 21st Century Cures Act provided $30 million in funding to the NIH over three years for clinical research for such therapies. However, these limited investments expire in fiscal year 2020.

The Alliance for Cell Therapy Now and the Regenerative Medicine Foundation support the recommendation of the authors, who urge funding for larger studies that build on the results to date. Collaboration and funding are also needed to collect and analyze the evidence from multiple ongoing and new studies, to better evaluate outcomes and potential benefits of MSC therapy for COVID-19 patients in need. A portion of the more than $10 billion in funding directed by Congress to the Biomedical Advanced Research and Development Authority (BARDA) and the NIH for COVID-19 should be used to support these goals.

About the Alliance for Cell Therapy Now

Alliance for Cell Therapy Now (ACT Now) is an independent, non-profit organization devoted to advancing the availability of and access to safe and effective cell therapies for patients in need. ACT Now convenes experts and stakeholders to develop and advance sound policies that will improve the development, manufacturing, delivery, and improvement of regenerative cell therapies. See http://allianceforcelltherapynow.org/

About the Regenerative Medicine Foundation

The non-profit Regenerative Medicine Foundation (RMF) fosters strategic collaborations to accelerate the development of regenerative medicine to improve health and deliver cures. RMF pursues its mission by producing its flagship World Stem Cell Summit, honoring leaders through the Stem Cell and Regenerative Medicine Action Awards, and promoting educational initiatives. STEM CELLS Translational Medicine is RMF's official journal partner. See https://www.regmedfoundation.org/

SOURCE Alliance for Cell Therapy Now

http://allianceforcelltherapynow.org

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MSC Therapy for Acute Respiratory Distress Syndrome; It's Time to Accelerate Clinical Trials for COVID-19 Patients in Need - PRNewswire

Groundbreaking Gene Therapies for Hereditary Diseases / Alessandro Aiuti, a physician and research scientist from Milan, receives the Else Krner…

The current coronavirus pandemic clearly illustrates how dangerous viral infections can become for us. Independent of the present situation, there are people whose bodies are defenseless against infections because their immune systems are unable to combat them - they suffer from immunodeficiency diseases such as ADA-SCID (adenosine deaminase severe combined immunodeficiency) or Wiskott-Aldrich syndrome. Prof. Dr. Alessandro Aiuti, a physician and research scientist based in Milan who works at the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) and at the Vita Salute San Raffaele University, is now being honored with the Else Krner Fresenius Prize for Medical Research 2020 for his groundbreaking successes in the development of gene therapies. The award is coupled to 2.5 million euros in prize money.

In the case of the rare immune disorder ADA-SCID, which exclusively afflicts young children and occurs about 15 times a year in Europe, a defective ADA gene within the genome disrupts lymphocyte development, leaving the young patient's body defenseless against infections. "Without effective therapy, the children rarely survive for more than 2 years because any infection can become perilous for them," Aiuti explains. Standard for this therapy is a bone marrow transplantation from a fully matched sibling. However, a suitable donor is available only for a minority of patients. "Meanwhile children with such a condition benefit from the advances we have made in the field of gene therapy. So far we have treated 36 children from 19 countries using the therapy we developed. In more than 80 percent of the cases, the treatment has had such an impact that no enzyme replacement therapy or transplantation is needed. This achievement has been made possible by the extraordinary effort and dedication of SR-Tiget researchers and clinical team throughout 25 years," Aiuti adds. All of the patients are still alive.

For these successes and his other work in the field of gene therapy, Alessandro Aiuti has now been honored with the Else Krner Fresenius Prize for Medical Research 2020 awarded by the Else Krner-Fresenius-Stiftung (EKFS) foundation. At 2.5 million euros, this award is one of the highest endowed prizes for medical research in the world. "Still young by comparison, this year the prize is being awarded for the third time. It honors research scientists for pioneering contributions in the areas of biomedical science. A major percentage of the prize money flows into the prizewinner's research and is supposed to contribute toward achieving further groundbreaking findings and medical breakthroughs in the future as well," emphasizes Prof. Dr. Michael Madeja, scientific director and member of the management board at EKFS.

The decision regarding the prize recipient was made by a ten-member international jury composed of renowned research scientists in the fields of genome editing and gene therapy along with delegates from the Scientific Commission at EKFS. Prof. Dr. Hildegard Bning, chairwoman of the jury and president of the European Society for Gene and Cell Therapy (ESGCT), substantiates the jury's decision: "Alessandro Aiuti is a truly outstanding physician and scientist. His work has decisively contributed to the development and successful treatment of rare, genetically caused disorders such as SCID. Thanks not least of all to the contributions he has made, even patients with other inheritable illnesses can presumably be treated successfully in the future."

After successful clinical trials, the gene therapy developed for ADA-SCID patients was approved as a pharmaceutical remedy in Europe. It is considered to be one of the key findings in the development of gene therapies worldwide. With this treatment certain blood stem cells (CD34+) are taken from the patient, then the cell DNA is modified. The cells are treated outside the body using a viral vector to accomplish this. The correct version of the gene for the ADA enzyme is introduced into the genome of the cells that were collected. The genetically modified cells are returned to the patient's bloodstream via intravenous infusion. A portion of the modified cells subsequently establish themselves in bone marrow again. The patient now has blood stem cells that function properly and produce lymphocytes to defend against infections - presumably on a life-long basis.

Alessandro Aiuti wants to utilize the prize money from EKFS to set the success story forth, to optimize the therapies further and map out the healing mechanisms involved in a better fashion. The scientist sees another major challenge in conveying the acquired knowledge beyond the successful gene therapies from Milan to as many other genetic disorders as possible. Alongside the therapy for ADA-SCID, the San Raffaele Telethon Institute for Gene Therapy has also developed gene therapies for four more hereditary diseases, among them the Wiskott-Aldrich syndrome and metachromatic leukodystrophy (MLD). To this day a total of more than 100 patients from 35 different countries have been treated.

Biography of Alessandro Aiuti

Alessandro Aiuti was born in Rome in 1966 and studied medicine there at Sapienza University. Following a stay at Harvard Medical School in Boston, Massachusetts in the USA, he received his doctorate in Human Biology in 1996 from Sapienza University. Since 1997 he has been active at the San Raffaele Scientific Institute in Milan, where he meanwhile also teaches as a professor at the Vita Salute San Raffaele University. He is furthermore Deputy Director of Clinical Research at the San Raffaele Telethon Institute for Gene Therapy and Head of the Pediatric Immunohematology Unit at San Raffaele Hospital.

Aiuti is the author of numerous and highly acclaimed publications. Over the course of his career he has received a number of prizes from national and international institutions. Aiuti is a member of the board of the European Society of Gene and Cell Therapy, and a member of the EMA Committee for Advanced Therapies since 2019.

The Else Krner Fresenius Prize for Medical Research

The international Else Krner Fresenius Prize for Medical Research came into existence in 2013 on the occasion of the 25th anniversary of Else Krner's death and is awarded in alternating fields of biomedical science. Endowed with 2.5 million euros, the prize is one of the most highly endowed medical research awards in the world. It honors and supports research scientists who have made significant scientific contributions in their fields and whose work can be expected to yield groundbreaking findings and medical breakthroughs in the future as well.

The Else Krner-Fresenius-Stiftung (EKFS) foundation - Advancing research. Helping people.

The Else Krner-Fresenius-Stiftung, a non-profit foundation, is dedicated to the funding of medical research and supports medical/humanitarian projects. The foundation was established in 1983 by entrepreneur Else Krner and appointed as her sole heir. EKFS receives virtually all of its income in dividends from the Fresenius healthcare group, in which the foundation is the majority shareholder. To date, the foundation has funded around 2,000 projects. With a current annual funding volume around 60 million euros the EKFS is one of the largest foundations for medicine in Germany. More information:www.ekfs.de.

The San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget)

Based in Milan, Italy, the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) is a joint venture between the Ospedale San Raffaele and Fondazione Telethon. SR-Tiget was established in 1995 to perform research on gene transfer and cell transplantation and translate its results into clinical applications of gene and cell therapies for different genetic diseases. Over the years, the Institute has given a pioneering contribution to the field with relevant discoveries in vector design, gene transfer strategies, stem cell biology, identity and mechanism of action of innate immune cells. SR-Tiget has also established the resources and framework for translating these advances into novel experimental therapies and has implemented several successful gene therapy clinical trials for inherited immunodeficiencies, blood and storage disorders, which have already treated >115 patients and have led through collaboration with industrial partners to the filing and approval of novel advanced gene therapy medicines.

Fondazione Telethon

Fondazione Telethon is a non-profit organisation created in 1990 as a response to the appeals of a patient association group of stakeholders, who saw scientific research as the only real opportunity to effectively fight genetic diseases. Thanks to the funds raised through the television marathon, along with other initiatives and a network of partners and volunteers, Telethon finances the best scientific research on rare genetic diseases, evaluated and selected by independent internationally renowned experts, with the ultimate objective of making the treatments developed available to everyone who needs them. Throughout its 30 years of activity, Fondazione Telethon has invested more than EUR 528 million in funding more than 2.630 projects to study more than 570 diseases, involving over 1.600 scientists. Fondazione Telethon has made a significant contribution to the worldwide advancement of knowledge regarding rare genetic diseases and of academic research and drug development with a view to developing treatments. For more information, please visit:www.telethon.it

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Groundbreaking Gene Therapies for Hereditary Diseases / Alessandro Aiuti, a physician and research scientist from Milan, receives the Else Krner...

Huge Growth Expected in Global Single-Cell Analysis Market in Future – Cole of Duty

The single-cell analysis market generated revenue of $1,688.9 million in 2015, and it is projected to grow at an 18.1% CAGR during the forecast period. The market is expected to be driven by the growing geriatric population, rising chronic disease prevalence, and increasing focus on personalized medicine. The study of individual cells isolated from multi-cellular organisms is referred to as single-cell analysis, which aims to study the heterogenous cell population. These studies are conducted in stem cell, neurological, cancer, and immunological research to help diagnose and treat various diseases.

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The single-cell analysis market is experiencing growth due to the rising healthcare expenditure across the globe. With the increasing prevalence of various diseases, governments of many countries are focusing more on improving the healthcare infrastructure. This fact has been agreed on by the World Health Organization, which reported that the total healthcare expenditure has witnessed an increase worldwide, thereby resulting in improvements in healthcare facilities. This is enabling people from different strata of the society in accessing healthcare facilities and tests involving single-cell analysis.

The geography segment of the single-cell analysis market is classified into Europe, Latin America, Asia, North America, and Rest of the World, among which the largest share in 2015 was accumulated by the North American region. Technical advancements in single-cell analysis products and growing geriatric population were instrumental in the North American markets growth. However, the fastest growth during the forecast period is expected to be witnessed by the Asian market, due to its large pool of patients and increasing awareness about new diagnostic techniques, such as single-cell analysis.

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GLOBAL SINGLE-CELL ANALYSIS MARKET

GLOBAL SINGLE-CELL ANALYSIS MARKET, BY TYPE OF PRODUCT

GLOBAL SINGLE-CELL ANALYSIS MARKET, BY TYPE OF CELL

GLOBAL SINGLE-CELL ANALYSIS MARKET, BY TYPE OF TECHNIQUE

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Huge Growth Expected in Global Single-Cell Analysis Market in Future - Cole of Duty